Novartis receives positive CHMP opinion for Kesimpta® (ofatumumab), a self-administered treatment for adult patients with relapsing multiple sclerosis with active disease

Novartis receives positive CHMP opinion for Kesimpta^® (ofatumumab), a self-administered treatment for adult patients with relapsing multiple sclerosis with active disease

•   CHMP opinion based on two Phase III ASCLEPIOS studies that met clinical and MRI endpoints, demonstrating a significant reduction in the number of relapses vs. teriflunomide, evaluated as the annualized relapse rate[i]

•   Relapsing forms of multiple sclerosis (RMS) is the most common form of MS, with around 85% of people in the UK considered to have relapsing remitting MS at the point of diagnosis[ii]

•   If approved, ofatumumab will be the first and only self-administered, targeted B-cell therapy to slow disease activity in patients living with RMS1

•   Self-administration at home gives RMS patients more flexibility and reduces the burden on patients, HCPs, and the NHS as it faces capacity challenges in light of COVID-19[iii]

London, January 29, 2021 — Novartis today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion and recommended granting marketing authorization of Kesimpta^® (ofatumumab) for the treatment of adult patients with relapsing forms of multiple sclerosis (RMS) with active disease, defined by clinical or imaging features. If approved, ofatumumab has the potential to become a first-choice treatment option for patients with RMS that can be self-administered once monthly at home via the Sensoready^® autoinjector pen¹.

Relapsing forms of MS (RMS) is the most common form, with around 85% of people considered to have relapsing remitting MS (RRMS) at the point of diagnosis². People living with RMS often experience patterns of new or worsening of old symptoms, lasting for a period of 24 hours or more[iv].

Despite the availability of a range of disease-modifying therapies (DMTs) for RMS, there are significant needs that are not fully met by current options. Research suggests that many individuals with RMS and on DMTs, continue to experience disease activity or safety and tolerability concerns[v]^,[vi].

“With relapsing forms of MS impacting the highest number of people living with MS in the UK, there is a need for a new treatment option that offers people with MS more flexibility and control in managing their disease outside of traditional hospital settings,” said Dr Wallace Brownlee, Consultant Neurologist and Clinical Lead for the Multiple Sclerosis Service at the National Hospital for Neurology and Neurosurgery. “Results from the ASCLEPIOS studies demonstrate that ofatumumab has a positive impact on reducing disease activity and slowing disability worsening in a convenient, once monthly formulation that can be given at home. Today’s news has the potential to offer the multiple sclerosis community, a much-needed self-administered treatment with proven benefits.”

“We are proud to be advancing innovative treatments that aim to positively impact people living with MS. Ofatumumab is a testament to our commitment to reimagine treatment in the early MS journey, and represents a significant step forward by delivering a new treatment that is effective and has a well-tolerated safety profile,” said Chinmay Bhatt, Managing Director UK, Ireland & Nordics for Novartis Pharmaceuticals. “As the NHS faces unprecedented challenges with capacity, at home treatments such as ofatumumab, have an added benefit in providing a valuable alternative option and reducing the burden on both NHS services and patients living with MS.”

The CHMP opinion is based on results from the Phase III ASCLEPIOS I and II studies, in which ofatumumab demonstrated superiority versus teriflunomide, a commonly prescribed oral disease modifying therapy for RMS. Results also showed a relative reduction in ARR by 50.5% (0.11 vs. 0.22) and 58.5% (0.10 vs. 0.25), compared with teriflunomide in ASCLEPIOS I and II respectively (P<0.001 in both studies)[vii]^,1. In additional post-hoc analysis, 87.8% (N=833) of patients treated with ofatumumab achieved no evidence of disease activity (NEDA-3) in their second year of treatment compared with 48.2% (N=821) of patients treated with teriflunomide (P<0.001)⁶. Consistent with the overall study population results, the post-hoc analysis from the Phase III ASCLEPIOS I and II trials analysis showed patients treated with ofatumumab experienced less frequent relapses, reduced inflammatory activity (evidenced by MRI scans), and prolonged time to disability worsening compared to those who received teriflunomide[viii].

Ofatumumab will be one of the first treatments to undergo the new regulatory process under new guidelines following the UK’s departure from the European Union. Novartis is committed to working with all UK stakeholders to bring ofatumumab to MS patients in the UK within the same timeframe as the EU. The UK Medicines and Healthcare products Regulatory Agency (MHRA) will consider the positive recommendation from the CHMP and is expected to deliver its final decision on the Marketing Authorisation Application (MAA) in line with EC Approval within a three-month timeframe.

About Kesimpta^® (ofatumumab)

Ofatumumab is a fully human anti-CD20 monoclonal antibody (mAb) in development for RMS that is self-administered by a once-monthly injection, delivered subcutaneously^1,[ix]. As shown in preclinical studies, ofatumumab is thought to work by binding to a distinct epitope on the CD20 molecule, inducing potent B-cell lysis and depletion[x]. Once-monthly dosing of ofatumumab allows faster repletion of B-cells versus other anti-CD20 monoclonal antibodies, and therefore may offer flexibility in the management of RMS⁷.

About ASCLEPIOS I and II studies

The ASCLEPIOS I and II studies are twin, identical design, flexible duration (up to 30 months), double-blind, randomized, multi-centre Phase III trials evaluating the safety and efficacy of ofatumumab versus teriflunomide in adults with RMS¹. The studies were conducted in 37 countries and enrolled 1,882 patients between the ages of 18 and 55 years, with an Expanded Disability Status Scale (EDSS) score between 0 and 5.5¹. Ofatumumab demonstrated a significant reduction in annualized relapse rate (ARR) by 50.5% (0.11 vs. 0.22) and 58.5% (0.10 vs. 0.25) compared with teriflunomide in ASCLEPIOS I and II, respectively (P<0.001 in both studies; primary endpoint)¹.

About ALITHIOS study

The ALITHIOS study is an ongoing, open-label, single arm, multi-centre extension Phase IIIb study evaluating the long-term safety, tolerability and effectiveness of ofatumumab in patients with RMS who have participated in prior ofatumumab studies. The primary endpoint is the number of patients that experience an adverse event or abnormal laboratory, vital and/or ECG results and positive suicidality outcomes. Secondary endpoints include number of relapses per year, 3- and 6-month CDW, 6-, 12- and 24-month confirmed disability improvement and improvement until end of study.

About APLIOS study

The APLIOS study is a 12-week, open-label, Phase II bioequivalence study to determine the onset of B-cell depletion with ofatumumab subcutaneous monthly injections and the bioequivalence of subcutaneous administration of ofatumumab via a prefilled syringe—as used in ASCLEPIOS I and II—and a Sensoready^® autoinjector pen in patients with RMS.

About Multiple Sclerosis
There are approximately 130,000 people with multiple sclerosis (MS) in the UK, and each year around 7,000 people are newly diagnosed with the condition[xi]. MS is a chronic disorder of the central nervous system (CNS) that disrupts the normal functioning of the brain, optic nerves and spinal cord through inflammation and tissue loss[xii]. The evolution of MS results in an increasing loss of both physical and cognitive functions (e.g. mobility problems, numbness, bladder and bowel problems, and problems with thinking, learning and planning)[xiii]. There are three types of MS: relapsing-remitting MS (RRMS), secondary progressive MS (SPMS) and primary progressive MS (PPMS)[xiv]. Patients with relapsing forms of MS – including RRMS and SPMS with active disease – experience distinct attacks of symptoms, known as relapses[xv],[xvi]. Around 85% of people are considered to have RRMS at their point of diagnosis[xvii]. SPMS, which typically follows from an initial RRMS course, is characterised by a gradual worsening of neurological function over time, and can be described as active (with relapses and/or evidence of new MRI activity) or not active (no evidence of current activity)^13,[xviii].

About Novartis in Multiple Sclerosis 

Novartis has a strong ongoing commitment to neuroscience and to bringing innovative treatments to patients suffering from neurological conditions where there is a high unmet need. The Novartis multiple sclerosis (MS) portfolio includes Gilenya^®▼ (fingolimod, an S1P modulator), which is licenced in Europe for the treatment of adults and children aged 10 and older with highly active relapsing remitting forms of MS. Mayzent^®▼ (siponimod) is licenced in Europe for the treatment of adult patients with SPMS with active disease evidenced by relapses or imaging features of inflammatory activity. Extavia^® (interferon beta-1b for subcutaneous injection) is approved in Europe to treat people with RRMS, people with SPMS with active disease (evidenced by relapses), and people who have had a single clinical event suggestive of MS.

About Novartis

Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach more than 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 130,000 people of nearly 150 nationalities work at Novartis around the world. Find out more at www.novartis.com.

In the UK, we employ approximately 1,500 people to serve healthcare needs across the whole of the UK, as well as supporting the global operations of Novartis. Since 2014, Novartis has invested over £200 million in R&D and is a leading sponsor of clinical trials, in the UK. For more information, please visit www.novartis.co.uk.

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